Pharmacy Prior Authorization Criteria

Kebilidi (eladocagene exuparvovec-tneq)

Indications for Prior Authorization

Kebilidi (eladocagene exuparvovec-tneq)

• For diagnosis of Aromatic L-amino acid decarboxylase (AADC) deficiency
  Indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.

Criteria

Kebilidi

Prior Authorization

Length of Approval: 1 Time Authorization in Lifetime

• Diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency
AND
• Disease is confirmed by one of the following:
• Molecular genetic testing confirms mutations in the DDC (DOPA decarboxylase) gene
OR
• CSF (cerebrospinal fluid) analysis showing a pattern of all of the following:
• Reduced levels of 5-hydroxyindoleacetic acid (5-HIAA), homovanillic acid (HVA) and 3-methoxy-4- hydroxyphenylglycol (MHPG)
• High concentrations of 3-O-methyldopa (3-OMD), L-Dopa, and 5- OH tryptophan (5-HTP)
• Normal pterins (e.g., neopterin, biopterin)
OR
• Analysis of plasma showing reduced aromatic L-amino acid decarboxylase (AADC) activity
AND
• Patient presents with the clinical signs and symptoms of the disease (e.g., hypotonia, oculogyric crises, developmental delays)
AND
• Assess skull maturity prior to the administration of Kebilidi [1]
AND
• Prescribed by a provider in a medical treatment center with expertise in gene therapy [A, 1, 7]
AND
• Prescribed by one of the following: [A, 1, 7]
• Neurologist
• Specialist with expertise in the diagnosis and management of aromatic L-amino acid decarboxylase (AADC) deficiency
AND
• Patient has never received Kebilidi treatment in their lifetime

P & T Revisions

2025-02-20

References

1. Kebilidi prescribing information. PTC Therapeutics, Inc. Warren, NJ. November 2024
2. ClinicalTrials.gov. A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants. Available at: https://www.clinicaltrials.gov/search?cond=NCT04903288. Accessed January 22, 2025.
3. AADC Insights. Accurate identification can help improve the care and management of patients with AADC deficiency1. Available at: https://aadcinsights.com/diagnosis/#:~:text=Current%20consensus%20guidelines%20recommend%20performing,a%20diagnosis%20of%20AADC%20deficiency. Accessed January 22, 2025.
4. Wassenberg, T., Molero-Luis, M., Jeltsch, K., et al. Consensus guideline for the diagnosis and treatment of aromatic l-amino acid decarboxylase (AADC) deficiency. Available at: https://ojrd.biomedcentral.com/articles/10.1186/s13023-016-0522-z. Accessed January 22, 2025.
5. Gantz, E., Sharer, J., McGrath, T., et al. Diagnosis of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency via Epilepsy Gene Panel Screening in a Patient with Atypical Presentation. Available at: https://journals.sagepub.com/doi/10.1177/2329048X231161027?icid=int.sj-full-text.similar-articles.1#:~:text=Current%20consensus%20guidelines%20describe%20three,(5%2DHIAA)%2C%20homovanillic. Accessed January 22, 2025.
6. Himmelreich, N., Montioli, R., Bertoldi, M., et al. Aromatic amino acid decarboxylase deficiency: Molecular and metabolic basis and therapeutic outlook. Available at: https://www.sciencedirect.com/science/article/abs/pii/S1096719218307868?via%3Dihub. Accessed January 22, 2025.
7. Andrews, R. Stereotactic neurosurgery. Available at: https://www.medlink.com/articles/stereotactic-neurosurgery. Accessed January 22, 2025.

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End Notes

1. The administration of Kebilidi involves stereotactic neurosurgery which involves using advanced imaging techniques (CT, MRI) and devices to translate the imaging information into guidance for the neurosurgical procedure. [7]

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Revision History

• 2025-02-20: New UM PA Guideline

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